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BioMarin gets go-ahead to start gene therapy clinical trials

15 Jan 2020

BMN 307 represents a potential third PKU treatment option from BioMarin and its second gene therapy clinical program.

BioMarin Pharmaceutical, a pioneer in treatments for the rare disease Phenylketonuria (PKU) and in gene therapy clinical research, has announced that both the FDA and the Medicines and Healthcare Products Regulatory Agency (MHRA) have granted the company Investigational New Drug (IND) status and approved its Clinical Trial Application (CTA), respectively, for its investigational gene therapy candidate BMN 307.

BMN 307 is an AAV5-phenylalanine hydroxylase (PAH) gene therapy designed to normalize blood phenylalanine (Phe) concentration levels in patients with PKU. BMN 307 will be evaluated to determine whether a single dose of treatment can restore natural Phe metabolism, normalize plasma Phe levels, and enable a normal diet in patients with PKU.

The company expects to start dosing patients in PHEARLESS, a Phase I/II study, in the first quarter of 2020 with product made at commercial scale from its award-winning gene therapy manufacturing facility. The company is actively preparing regulatory submissions to open additional clinical sites in other countries. BMN 307 represents a potential third PKU treatment option from BioMarin and its second gene therapy clinical program. Both the FDA and European Medicines Agency (EMA) have granted BMN 307 Orphan Status.

BMN 307 follows BioMarin's first investigational gene therapy clinical program: valoctocogene roxaparvovec to treat severe hemophilia A, currently in Phase III. Based on data from an interim analysis of the ongoing Phase III study, the EMA validated a Marketing Authorization Application in the EU for valoctocogene roxaparvovec, and the review process has begun. The company also has submitted a Biologics License Application to the FDA and anticipates the review to begin in February 2020.

"With BMN 307, we are joining together our expertise in PKU biology and the knowledge we have gained from developing the only two approved therapies for PKU with our understanding of gene therapy clinical development and manufacturing from our valoctocogene roxaparvovec experience," said Hank Fuchs, President, Worldwide Research and Development at BioMarin. "BioMarin has stood with the PKU community for over 15 years and remains dedicated to continuing to increase the body of medical knowledge in this devastating disease."

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